(News Bulletin 247) – The company indicated that its American partner has successfully produced a first batch of Lumevoq that complies with good manufacturing practice standards. The title soars on the Paris Stock Exchange.

GenSight announces good news for Lumevoq, its cell therapy aimed at correcting a degenerative disease responsible for sudden blindness. The biopharmaceutical company specializing in gene therapies to treat neurodegenerative diseases of the retina and central nervous system, jumped another 65% after surging 90% Monday morning around 10:00 a.m. A classic reaction for a “penny stock”, a value whose price is less than one euro and which is subject to high volatility.

The French pioneer of cell therapies targeting vision diseases is therefore sought after on the Paris Stock Exchange while its production partner in the United States has successfully produced a batch of Drug Substance (DS) for Lumevoq that complies with good standards. manufacturing practices (“Good Manufacturing Practice, GMP”).

Lumevoq is GenSight’s lead product candidate for the treatment of Leber’s hereditary optic neuropathy (LHON). This genetic disease which mainly affects young men and which results in a sudden and rapid loss of vision without causing total blindness.

GenSight Biologics explains that the batch in question was manufactured in accordance with GMP manufacturing protocols, which are the standards required for commercial batches. The company adds that this “gene therapy can be administered to patients after having successfully passed all quality control tests, and pending approval from regulatory bodies”.

A success which “clarifies the calendar for Lumevoq”

GenSight Biologics also says a second batch of Drug Substance is in production. It will aim to provide, according to the company, more doses available to patients and the “viral titer” results are expected next October, adds GenSight.

In total, GenSight plans to produce 3 batches compliant with good manufacturing practice standards using the commercial manufacturing process, but outside the context of a validation campaign.

A validation campaign (Process Performance Qualification or PPQ) consists of sequentially and successfully manufacturing a minimum of 3 GMP (Good Manufacturing Practices) batches, standards required for human use outside of a clinical trial. ) in order to demonstrate and document the robustness, control, consistency and reproducibility of the commercial manufacturing process within the selected commercial production site

GenSight Biologics wishes to acquire more batch production data to strengthen a future marketing authorization application (MAA) submission as well as more experience with the manufacturing process for operational teams. The results are expected in September and October 2023.

The goal being for the company to respond “to the immediate need to supply product” both to launch a possible new clinical trial, and for a possible resumption of the early access program in the first quarter of 2024.

“Lumevoq should again be available to patients and doctors in early 2024. This success also clarifies our timetable for Lumevoq, which is excellent news for patients suffering from LHON and blinded by the disease,” commented Bernard Gilly, co-founder and CEO of GenSight Biologics.

Cascading delays

It must be said that Gensight’s stock market performance has been penalized by the successive delays in the release of Luvemoq, which is likely to fuel “a feeling of weariness among investors”, underlined Oddo in a note last March. Despite today’s increase, GenSight still shows a decline of more than 80% since the start of the year.

Last spring, Gensight made the decision to abandon the process to obtain marketing authorization for Lumevoq from the European Medicines Agency. The company hoped to obtain a green light on European soil at the end of the year for its treatment.

“GenSight must now identify the best way to bring its product to market but it seems obvious to us that this will require new clinical studies, which will result in additional delays and costs,” Degroof Petercam said in reaction to this news. The design office then placed its advice under review after this disappointment. Lumevoq constituted 90% of the company’s value in the design office’s valuation.

Visibility still low

In this context, the company had pressing short-term financing needs. In April, it estimated that it had sufficient working capital to meet its financial obligations until June. As of June 30, the company’s available cash amounted to 1 million euros according to the latest results published by the company. An insufficient amount to finance the rest of the operations.

To preserve its cash flow, GenSight, for example, reduced its workforce by 40%, mainly in the sales teams. The company was also able to recover its 2022 Research Tax Credit, amounting to 2.2 million euros, in July 2023.

Above all, the company benefited from a valuable financial boost this summer. GenSight’s cash horizon has been extended after obtaining new financing of 10 million euros last August from Sofinnova Partners, Invus and UPMC Enterprises. It is broken down into two tranches of 6 and 4 million euros.

The company subsequently drew down the first tranche in the form of convertible bonds, allowing GenSight to be financed until October. And it is thanks to the net proceeds from this issue that the company was able to launch the start of production of batches complying with good manufacturing practice standards.

As for the second tranche, this remains conditional on the production of at least two successive GMP (Good Manufacturing Practice) batches of Lumevoq, or the unanimous approval of the three investors, no later than October 30, 2023. If GenSight meets the requirements of its financial backers, the company will be awarded a new tranche of 4 million euros, allowing it to extend its cash horizon until December 2023.

Moving forward, the company plans to seek other sources of financing in debt or equity to be able to guarantee the continuation of its operations beyond the end of 2023. At the same time, GenSight says it is in active discussions “with potential partners on strategic opportunities, including merger or acquisition”, which are evaluated as one of the options considered by GenSight to continue its adventure.

“The return of the European Medicines Agency (EMA) will be decisive in defining the future of Lumevoq, knowing that the EMA report could make it possible to extend funding beyond 2024,” indicates Degroof Petercam in his note of the day. The analysts maintain their opinion “under review”, while waiting to have “more visibility” on Lumevoq.